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- W2788397742 abstract "Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spinal muscular atrophy, Duchenne muscular dystrophy and familial amyloid polyneuropathy to highlight the different mechanisms of action of ASOs and RNAi." @default.
- W2788397742 created "2018-03-06" @default.
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- W2788397742 date "2018-02-17" @default.
- W2788397742 modified "2023-09-24" @default.
- W2788397742 title "Antisense oligonucleotides and other genetic therapies made simple" @default.
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- W2788397742 doi "https://doi.org/10.1136/practneurol-2017-001764" @default.
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